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Evaluating axatilimab and corticosteroids for treating chronic graft-versus-host disease

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease (AXemplify-357)

Phase 3 Interventional Incyte Corporation · NCT06585774

This study is testing if adding a new drug called axatilimab to standard steroids can help people aged 12 and older who have recently developed moderate to severe chronic graft-versus-host disease feel better.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment240 (estimated)
Ages12 Years and up
SexAll
SponsorIncyte Corporation Industry-sponsored
Drugs / interventionsprednisone, axatilimab
Locations119 sites (Birmingham, Alabama and 118 other locations)
Trial IDNCT06585774 on ClinicalTrials.gov

What this trial studies

This study aims to compare the effectiveness of axatilimab, an investigational drug, against a placebo when used alongside corticosteroids for the initial treatment of moderate to severe chronic graft-versus-host disease (cGVHD). Participants will include individuals aged 12 and older who have recently developed cGVHD and require systemic therapy. The study will assess the outcomes of these treatments to determine if axatilimab provides a significant benefit over standard care. The trial will be conducted across multiple locations in California and Florida.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 12 and older with new-onset moderate or severe cGVHD requiring systemic therapy.

Not a fit: Patients who have received more than one prior allo-HCT or have overlap cGVHD may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could offer a more effective option for patients suffering from chronic graft-versus-host disease.

How similar studies have performed: Other studies have explored treatments for cGVHD, but the specific combination of axatilimab and corticosteroids is a novel approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* ≥ 12 years of age at the time of informed consent.
* New-onset moderate or severe cGVHD, as defined by the 2014 NIH Consensus Development Project Criteria for Clinical Trials in cGVHD, requiring systemic therapy.
* History of allo-HCT from any donor HLA type (related or unrelated donor with any degree of HLA matching) using any graft source (bone marrow, peripheral blood stem cells, or cord blood). Recipients of myeloablative, nonmyeloablative, or reduced-intensity conditioning are eligible.
* Adequate hematologic function with ANC ≥ 0.5 × 109/L independent of growth factors for at least 7 days prior to study entry.
* Willingness to avoid pregnancy or fathering children.

Exclusion Criteria:

* Received more than 1 prior allo-HCT. Prior autologous HCT is allowed.
* Has overlap cGVHD, defined as simultaneous presence of features or characteristics of aGVHD in a patient with cGVHD.
* Received more than 7 days of systemic corticosteroid treatment for cGVHD or unable to begin a prednisone dose ≥ 1.0 mg/kg per day (or methylprednisolone equivalent) for cGVHD.
* Received previous systemic treatment for cGVHD, including extracorporeal photopheresis.
* Systemic treatment with CNIs or mTOR inhibitors started within 2 weeks prior to C1D1.
* Prior treatment with CSF-1R targeted therapies.
* Active, uncontrolled bacterial, fungal, parasitic, or viral infection.
* Evidence of relapse of the primary hematologic disease or treatment for relapse after the allo-HCT was performed, including DLIs for the treatment of molecular relapse.
* History of acute or chronic pancreatitis.
* Active symptomatic myositis.
* History or current diagnosis of cardiac disease indicating significant risk of safety for participation in the study, such as uncontrolled or significant cardiac disease.
* Severe renal impairment, that is, estimated CrCl \< 30 mL/min measured or calculated by Cockcroft-Gault equation in adults and Schwartz formula in pediatric participants, or endstage renal disease on dialysis.
* Impaired liver function, defined as total bilirubin \> 1.5 × ULN and/or ALT and AST \> 3 × ULN in participants with no evidence of liver cGVHD.
* Pregnant or breastfeeding.

Other protocol-defined Inclusion/Exclusion Criteria may apply.

Where this trial is running

Birmingham, Alabama and 118 other locations

+69 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Chronic Graft-versus-host-diseasecGVHD
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.